We saw continued growth and pipeline progress in the third quarter of 2021, and it is satisfying to see that results are confirming our strategic direction. Overall, our two main business areas are in solid shape and they progressed as expected

With the goal of advancing care and providing innovative treatment approaches for people living with rare haematologic diseases, we are excited to share data at this year’s virtual ISTH

We are committed to transforming the lives of people living with rare diseases; the breadth of our activity and our presentations at EHA are important contributions to expanding the understanding and knowledge of the community in support of our commitment

The PRINCE study results reinforce the efficacy and safety profile of pegcetacoplan in PNH

The COVID-19 pandemic, with its related restrictions and lockdowns, has continued to impact our markets and operations. I believe that we will be able to recover many of the adverse effects of COVID-19 over time. Despite this unfavourable environment we have been able to strategically progress our core portfolio. We have advanced key products, such as pegcetacoplan and efanesoctocog alfa, in our R&D portfolio, and we are exploring new opportunities for anakinra for the treatment of hyperinflammation related to COVID-19.

In 2020, we continued to take major steps in the transformation of the company and expanded beyond our traditional stronghold in haemophilia. We achieved solid growth during the year, and broadened the foundation of the company to align with our ambition of becoming a global leader in rare disease

2020 was a year of extreme challenges but also great opportunities. The COVID-19 pandemic created a challenging situation for the whole organisation, which did its utmost to serve customers and ensure supply to patients. At the same time, we entered two important strategic partnerships fuelling our pipeline, strengthening our international footprint and enabling strong future growth. We closed the full year 2020 with solid revenue of SEK 15,261 M, both core areas showing double-digit growth. Adjusted EBITA grew by 3 per cent to SEK 6,301 M.

Today’s announcement marks an important step for those affected by primary HLH, as currently there are no approved treatments available in China to meet the vast unmet medical need for this life threatening condition

Sobi has been through a significant transformation in the past few years and we are now well positioned for the next phase

We are delighted that the first patient in the phase 2 clinical study has been dosed as there is an urgency for a treatment for patients with ALS. In collaboration with Apellis, we look forward to evaluating the potential of pegcetacoplan in patients with ALS.

This recommendation by the CHMP is disappointing given the significant unmet medical need which exists for patients with pHLH who have no approved therapies in Europe. During the re-examination we worked extensively with physicians and patients and were able to resolve some but not all of the concerns raised by EMA

The launch of Doptelet offers an additional treatment option for patients living with CLD. Since the acquisition of Dova Pharmaceuticals, our focus has been on expanding sustainable patient access to Doptelet from the US to Europe. We are delighted that Doptelet is now available in the UK for this initial indication, and we look forward to continue rolling out Doptelet in other European markets

This analysis confirms the methodology and use of clinically objective response criteria in the pivotal study assessing treatment efficacy of emapalumab in primary HLH, where there are currently no validated endpoints for efficacy assessment. We remain committed to making emapalumab accessible to patients affected by this severe condition around the world.

While we are disappointed that avatrombopag failed to show efficacy in CIT, we are deeply grateful to the patients, investigators, and clinical site staff who contributed to the study’s completion. Due to the lack of an approved treatment we firmly believe that avatrombopag could benefit patients with CIT. We were pleased to see that few patients required platelet transfusions or dose modifications in this study and were able to receive their chemotherapy without interruption

SEL-212 is a highly differentiated product candidate for the treatment of chronic refractory gout, and these data reinforce our excitement about its potential. We are proud to collaborate with Selecta for the ongoing phase 3 programme which has already enrolled the first patient

SEL-212 has the potential to significantly improve outcomes for patients with chronic refractory gout, and we are proud to have taken the next step towards making this therapy accessible to patients unable to maintain treatment with the currently available options.

We are very proud of the collaboration between Sobi and the DevelopAKUre partners, especially the AKU Society, which has resulted in today’s announcement. This recommendation, if approved by the European Commission, would help address a medical need that has been unmet for people with AKU since the discovery of the condition more than 120 years ago.

"Factor VIII replacement therapy remains a cornerstone of care in haemophilia A and is a single therapy that can be used across numerous treatment scenarios including, prophylaxis, acute bleed control and perioperative management"

Emapalumab has demonstrated a positive benefit/risk profile in primary HLH in a post-approval real life setting in the US since the FDA approval in 2018. The product has been able to make a substantial difference for a very vulnerable group of patients in the US. We will do our utmost to share these insights and address the open questions by CHMP during the re-examination with a view to secure access for primary HLH in children to this treatment in Europe.

The COVID-19 pandemic has continued to pose challenges for the whole world and has of course had implications for Sobi as well. However, we have made significant efforts and have made strategic progress in this difficult environment

These are the first published final results for extended half-life (EHL) treatments in PUPs demonstrating tolerance and efficacy in this vulnerable patient population.

Today’s announcement is exciting news for the haemophilia A community in the UK. The agreement ensures continued and increased access to Elocta for people living with haemophilia A of all ages and all severities

For lasting change to become a reality, we need to recognise that access to treatment is a fundamental human right. We are proud to do our part to address this critical health issue, in partnership with Sanofi

SEL-212 is a unique, late-stage product candidate containing a novel enzyme and a potentially ground-breaking immune tolerance platform, which could significantly improve the treatment for patients with chronic refractory gout, a debilitating disease with significant unmet need

The publication of the results in this highly respected medical journal is a testament to the medical importance of the emapalumab findings for patients with primary HLH.

Total revenue for the first quarter 2020 was SEK 4,639 M, up 42 per cent year-on-year, while EBITA was SEK 2,173 M. The COVID-19 pandemic posed challenges for Sobi, as for the rest of the world, during the first quarter.

The COVID-19 global pandemic requires everyone to play their part. We believe it is our duty to support this important request and will focus our attention on this exploratory study.

Our growth journey continued in the third quarter, strengthening our Haematology franchise with the announcements of the intention to acquire Dova Pharmaceuticals1and our decision to opt in early to the development of BIVV001. Revenue growth in the quarter was 27 per cent with revenue of SEK 2,930 M (2,315). EBITA was SEK 1,099 M, resulting in an EBITA margin of 38 per cent

The cadence of upcoming launches and approvals across indications and regions that Doptelet provides, enables us to further accelerate growth in our haematology franchise

Our strong performance continued in the second quarter, with organic growth of 25 per cent taking us to revenue of SEK 3,163 M, adjusted EBITA of SEK 1,193 M and an adjusted EBITA margin of 38 per cent. Announcing the acquisition of emapalumab and related assets, we took further steps to sharpen focus on our core therapeutic areas and on late-stage assets

We are proud to support Investigator-Sponsored Studies because the research conducted by the community adds substantially to an increased understanding of pre-clinical and clinical value of haemophilia treatment

This data supports the observation that the introduction of EHLs has allowed for people with haemophilia to live a life beyond haemophilia, with increased quality of life and a greater ability to engage in sports and activities

Development of inhibitors continues to be a significant challenge for people with haemophilia and it remains an area of unmet need

Sobi is committed to liberating life for people with haemophilia. These results suggest that even in a country such as Sweden, with well-established use of prophylactic treatment and high levels of access to treatment, there is a need for further research to address knowledge gaps and enable people to live a life beyond haemophilia

By increasing the scientific evidence and the understanding of the clinical value of our extended half-life products in all patient groups living with haemophilia, we maintain our focus on research and an approach to treatment that reflects a meaningful difference for patients, allowing them to live a life beyond haemophilia

It is extremely encouraging to see a complete response in the first six patients treated with emapalumab, particularly considering that MAS is a serious and potentially fatal complication, and that all of these patients had failed other treatments

Sobi’s position is stronger than ever and I see ample opportunity to take Sobi to the next level.

It is of great importance to offer an individualised prophylactic treatment to reduce the need for and occurrence of surgery as well as an optimal and measurable factor treatment during surgery.

“We started 2019 with a strong first quarter, with revenues of SEK 3,265 M and EBITA of SEK 1,509 M, corresponding to an EBITA margin of 46 per cent. We completed the acquisition of Synagis in late January and are in the process of integrating Synagis operations and employees into the organisation. The Haemophilia franchise continued to grow strongly, we have seen positive effects from the Gamifant launch in the US even though it is still in early launch phase, and we see an increasing demand for Synagis.”

2018 was a year of transformation for Sobi. Our substantial progress in Haemophilia, acquisitions in Immunology and sales growth in Specialty Care mean that the company is essentially, on a pro-forma basis, two-and-a-half times the size it was at the end of 2016. But it is important to understand that we see this as an important stepping stone. In the Annual and Sustainability Report, we outline how the strong growth we delivered throughout 2018 is fuelling our vision of becoming global leaders in providing innovative treatments that transform life for people with rare diseases

“Liberate Life not only spells out our vision for the future of haemophilia care but also serves as a call to action for involvement from patients, advocates and healthcare professionals.”

We are honoured to be recognised by NORD for the work we have done to bring Gamifant to primary HLH patients. Primary HLH is often fatal, and we are grateful to be able to contribute to helping these very sick patients.

In this study, the majority of these very sick patients who were treated with emapalumab were able to reach transplant, engraft and survive

2018 was a significant year for Sobi. Revenues grew 40 per cent, totalling SEK 9,139 M (6,511) for the full year. We generated operating leverage and grew earnings by 74 per cent, with EBITA of SEK 3,571 M (2,053). Gross margin rose to 74 per cent (72).

By increasing the scientific evidence and the understanding of the clinical value of our extended half-life products in all patient groups living with haemophilia, we maintain our focus on research and an approach to treatment that reflects a meaningful difference for patients, allowing them to live a life beyond haemophilia

“This new expanded indication allows all patients access to treatment, which is particularly significant as the disease often presents in newborns,” says Norbert Oppitz, Head of Specialty Care at Sobi. “Following stabilisation with acute intravenous therapy, children can now be managed long-term on Ravicti in the early days of their lives.”

We are very encouraged by the data being presented at ASH on emapalumab in primary HLH, and we look forward to investigating emapalumab in additional diseases for which interferon gamma is considered pathogenic

Joint protection remains a significant challenge in the long-term treatment of haemophilia keeping individuals from living a life without constraints of their disease and these results confirm that our therapies can play a role in the effective prevention of joint bleeds

We are thrilled that the clinical data on emapalumab in primary HLH has been selected for presentation at this important medical congress. We appreciate this recognition from ASH as it confirms the relevance of the underlying science to further the understanding of HLH, and the spotlight this shines on a very rare life-threathening disease. This would not have been possible without the support of the study investigators and participanting patients over a number of years

The FDA approval of Gamifant marks an important milestone in Sobi’s vision to bring transformative treatments to patients with a high medical need,